Below are the first 10 and last 10 pages of uncorrected machine-read text (when available) of this chapter, followed by the top 30 algorithmically extracted key phrases from the chapter as a whole.
Intended to provide our own search engines and external engines with highly rich, chapter-representative searchable text on the opening pages of each chapter.
Because it is UNCORRECTED material, please consider the following text as a useful but insufficient proxy for the authoritative book pages.
Do not use for reproduction, copying, pasting, or reading; exclusively for search engines.
OCR for page R1
Improving the Quality of Cancer Clinical Trials: Workshop Summary
IMPROVING THE QUALITY OF CANCER CLINICAL TRIALS
WORKSHOP SUMMARY
National Cancer Policy Forum
Margie Patlak and Sharyl Nass, Rapporteurs
INSTITUTE OF MEDICINE OF THE NATIONAL ACADEMIES
THE NATIONAL ACADEMIES PRESS
Washington, D.C.
www.nap.edu
OCR for page R2
Improving the Quality of Cancer Clinical Trials: Workshop Summary
THE NATIONAL ACADEMIES PRESS
500 Fifth Street, N.W. Washington, DC 20001
NOTICE: The project that is the subject of this report was approved by the Governing Board of the National Research Council, whose members are drawn from the councils of the National Academy of Sciences, the National Academy of Engineering, and the Institute of Medicine.
This study was supported by Contract Nos. HHSN261200611002C, 200-2005-13434 TO #1, HHSP233200700373P, and 223-01-2460 TO #27, between the National Academy of Sciences and the National Cancer Institute, the Centers for Disease Control, the Agency for Healthcare Research and Quality, and the Food and Drug Administration, respectively. This study was also supported by the American Cancer Society, the American Society for Clinical Oncology, C-Change and the Association of American Cancer Institutes. Any opinions, findings, conclusions, or recommendations expressed in this publication are those of the author(s) and do not necessarily reflect the view of the organizations or agencies that provided support for this project.
International Standard Book Number-13: 978-0-309-11668-8
International Standard Book Number-10: 0-309-11668-6
Additional copies of this report are available from the
National Academies Press,
500 Fifth Street, N.W., Lockbox 285, Washington, DC 20055; (800) 624-6242 or (202) 334-3313 (in the Washington metropolitan area); Internet, http://www.nap.edu.
For more information about the Institute of Medicine, visit the IOM home page at: www.iom.edu.
Copyright 2008 by the National Academy of Sciences. All rights reserved.
Printed in the United States of America
The serpent has been a symbol of long life, healing, and knowledge among almost all cultures and religions since the beginning of recorded history. The serpent adopted as a logotype by the Institute of Medicine is a relief carving from ancient Greece, now held by the Staatliche Museen in Berlin.
Suggested citation: IOM (Institute of Medicine). 2008. Improving the quality of cancer clinical trials: Workshop summary. Washington, DC: The National Academies Press.
OCR for page R3
Improving the Quality of Cancer Clinical Trials: Workshop Summary
“Knowing is not enough; we must apply.
Willing is not enough; we must do.”
—Goethe
INSTITUTE OF MEDICINE OF THE NATIONAL ACADEMIES
Advising the Nation. Improving Health.
OCR for page R4
Improving the Quality of Cancer Clinical Trials: Workshop Summary
THE NATIONAL ACADEMIES
Advisers to the Nation on Science, Engineering, and Medicine
The National Academy of Sciences is a private, nonprofit, self-perpetuating society of distinguished scholars engaged in scientific and engineering research, dedicated to the furtherance of science and technology and to their use for the general welfare. Upon the authority of the charter granted to it by the Congress in 1863, the Academy has a mandate that requires it to advise the federal government on scientific and technical matters. Dr. Ralph J. Cicerone is president of the National Academy of Sciences.
The National Academy of Engineering was established in 1964, under the charter of the National Academy of Sciences, as a parallel organization of outstanding engineers. It is autonomous in its administration and in the selection of its members, sharing with the National Academy of Sciences the responsibility for advising the federal government. The National Academy of Engineering also sponsors engineering programs aimed at meeting national needs, encourages education and research, and recognizes the superior achievements of engineers. Dr. Charles M. Vest is president of the National Academy of Engineering.
The Institute of Medicine was established in 1970 by the National Academy of Sciences to secure the services of eminent members of appropriate professions in the examination of policy matters pertaining to the health of the public. The Institute acts under the responsibility given to the National Academy of Sciences by its congressional charter to be an adviser to the federal government and, upon its own initiative, to identify issues of medical care, research, and education. Dr. Harvey V. Fineberg is president of the Institute of Medicine.
The National Research Council was organized by the National Academy of Sciences in 1916 to associate the broad community of science and technology with the Academy’s purposes of furthering knowledge and advising the federal government. Functioning in accordance with general policies determined by the Academy, the Council has become the principal operating agency of both the National Academy of Sciences and the National Academy of Engineering in providing services to the government, the public, and the scientific and engineering communities. The Council is administered jointly by both Academies and the Institute of Medicine. Dr. Ralph J. Cicerone and Dr. Charles M. Vest are chair and vice chair, respectively, of the National Research Council.
www.national-academies.org
OCR for page R5
Improving the Quality of Cancer Clinical Trials: Workshop Summary
WORKSHOP PLANNING COMMITTEE
JOHN MENDELSOHN, M.D. (Co-Chair), President,
MD Anderson Cancer Center
HAROLD MOSES, M.D. (Co-Chair), Professor of Molecular Oncology, Cancer Biology, Medicine, and Pathology; Director Emeritus,
Vanderbilt-Ingram Cancer Center, Vanderbilt University Medical Center
JAMES DOROSHOW, M.D., Director,
Division of Cancer Treatment and Diagnosis, National Cancer Institute
ROY HERBST, M.D., Ph.D., Professor and Chief,
Thoracic/Head and Neck Medical Oncology, MD Anderson Cancer Center
HEDVIG HRICAK, M.D., Professor and Chair,
Department of Radiology, Memorial Sloan-Kettering Cancer Center
GILBERT OMENN, M.D., Ph.D., Professor,
Internal Medicine, Human Genetics, and Public Health, University of Michigan Medical School
DAVID PIWNICA-WORMS, M.D., Ph.D., Professor,
Department of Radiology, Washington University School of Medicine
JOHN WAGNER, M.D., Ph.D., Executive Director,
Department of Clinical Pharmacology, Merck, Inc.
JANET WOODCOCK, M.D., Deputy Commissioner and Chief Medical Officer,
Food and Drug Administration
IOM planning committees are solely responsible for organizing the workshop, identifying topics, and choosing speakers. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution.
OCR for page R6
Improving the Quality of Cancer Clinical Trials: Workshop Summary
NATIONAL CANCER POLICY FORUM
HAROLD L. MOSES, M.D. (Chair), Professor of Molecular Oncology, Cancer Biology, Medicine, and Pathology; Director Emeritus,
Vanderbilt-Ingram Cancer Center, Vanderbilt University Medical Center
PETER BACH, M.D., M.A.P.P., Associate Attending Physician,
Memorial Sloan-Kettering Cancer Center
EDWARD BENZ, JR., M.D., President,
Dana-Farber Cancer Center
THOMAS BURISH, Ph.D., Past Chair,
American Cancer Society Board of Directors, and
Provost,
Notre Dame University
BETTY FERRELL, Ph.D., R.N., F.A.A.N., Research Scientist,
City of Hope National Medical Center
JOSEPH FRAUMENI, JR., M.D., Director,
Division of Cancer Epidemiology and Genetics, National Cancer Institute
STEPHEN FRIEND, M.D., Ph.D., Executive Vice President,
Oncology, Merck, Inc.
PATRICIA GANZ, M.D.,
American Society of Clinical Oncology,
Board Member, and Professor of Medicine,
University of California, Los Angeles
ROBERT GERMAN, Dr.Ph., M.P.H., Associate Director for Aging,
Division of Cancer Prevention and Control, Centers for Disease Control and Prevention
ROY HERBST, M.D., Professor and Chief,
Thoracic/Head and Neck Medical Oncology, MD Anderson Cancer Center
THOMAS KEAN, M.P.H., Executive Director,
C-Change
WILLIAM LAWRENCE, M.D., M.S., Medical Director,
Center for Outcomes and Evidence, Agency for Healthcare Research and Quality
LYNN MATRISIAN, Ph.D., Professor and Chair,
Department of Cancer Biology, Vanderbilt-Ingram Cancer Center, Vanderbilt University School of Medicine
JOHN NIEDERHUBER, M.D., Director,
National Cancer Institute
DAVID PARKINSON, M.D., Senior Vice President,
Oncology Research and Development, Biogen IDEC
EDITH PEREZ, M.D., Professor of Medicine and Director,
Cancer Clinical Study Unit, Mayo Clinic
SCOTT RAMSEY, M.D., Ph.D., Full Member,
Fred Hutchinson Cancer Research Center
OCR for page R7
Improving the Quality of Cancer Clinical Trials: Workshop Summary
MARGARET SPITZ, M.D., Chair of Epidemiology,
MD Anderson Cancer Center
ELLEN STOVALL, President and Chief Executive Officer,
National Coalition for Cancer Survivorship
BARRY STRAUBE, M.D., Director,
Office of Clinical Standards and Quality, Centers for Medicare & Medicaid Services
JANET WOODCOCK, M.D., Deputy Commissioner and Chief Medical Officer,
Food and Drug Administration
Staff
SHARYL NASS, Ph.D., Study Director
ROGER HERDMAN, M.D., Director,
National Cancer Policy Forum
LAURA LEVIT, J.D., Research Assistant
CHRISTINE MICHEEL, Ph.D., Christine Mirzayan Science and Technology Policy Graduate Fellow
MICHAEL PARK, Senior Program Assistant
ALEX REPACE, Senior Program Assistant
MARGIE PATLAK, Science Writer
IOM forums and roundtables do not issue, review, or approve individual documents. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution.
OCR for page R8
Improving the Quality of Cancer Clinical Trials: Workshop Summary
This page intentionally left blank.
OCR for page R9
Improving the Quality of Cancer Clinical Trials: Workshop Summary
Reviewers
This report has been reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise, in accordance with procedures approved by the National Research Council’s Report Review Committee. The purpose of this independent review is to provide candid and critical comments that will assist the institution in making its published report as sound as possible and to ensure that the report meets institutional standards for objectivity, evidence, and responsiveness to the study charge. The review comments and draft manuscript remain confidential to protect the integrity of the deliberative process. We wish to thank the following individuals for their review of this report:
Ray DuBois, M.D., Ph.D., University of Texas MD Anderson Cancer Center
Doug Hanahan, Ph.D., University of California, San Francisco
Hedvig Hricak, M.D., Ph.D., Memorial Sloan-Kettering Cancer Center
Susan M. Jerian, M.D., OncoRD, Inc.
Daniel C. Sullivan, M.D., Duke Cancer Center
Although the reviewers listed above have provided many constructive comments and suggestions, they were not asked to endorse the final draft of the report before its release. The review of this report was overseen
OCR for page R10
Improving the Quality of Cancer Clinical Trials: Workshop Summary
by Melvin Worth, M.D. Appointed by the Institute of Medicine, he was responsible for making certain that an independent examination of this report was carried out in accordance with institutional procedures and that all review comments were carefully considered. Responsibility for the final content of this report rests entirely with the authors and the institution.
OCR for page R11
Improving the Quality of Cancer Clinical Trials: Workshop Summary
Contents
Improving the Quality of Cancer Clinical Trials: Workshop Summary
1
Introduction,
1
New Clinical Trial Designs,
3
Phase 0 Trials,
3
Adaptive Trial Designs,
16
Targeting Multiple Pathways with Multiple Drugs,
23
Preclinical Model Systems,
36
Molecular Imaging,
40
Current and Developing Methods,
40
Challenges of Molecular Imaging,
46
Screening for Predictive Markers,
49
The Challenges of Clinical Validation,
50
Bioimaging Predictive Markers,
55
Clinical Translation,
59
Panel Discussion,
65
Costs of Clinical Trials,
67
Regulatory Costs,
68
Patient Accrual,
70
Global Outsourcing,
72
Time Is Money,
74
Public–Private Collaborations,
75
OCR for page R12
Improving the Quality of Cancer Clinical Trials: Workshop Summary
Regulatory Issues,
77
Regulatory Barriers to Innovation,
77
Patient Advocacy Perspective,
81
Regulation of In Vitro Diagnostics,
83
Regulatory Issues in Improving Cancer Clinical Trials,
86
Reports from the Case Study Discussion Groups,
89
Adaptive Trial Design,
89
Phase 0 Trials,
91
Imaging,
93
Use of Proteomics/Genomics to Assign Therapy in Lung Cancer,
96
Use of Genetics/Genomics to Assign Therapy,
98
Concluding Remarks,
101
References,
102
Acronyms
107
Glossary
109
Appendixes
A
Workshop Agenda
115
B
Workshop Speakers, Moderators, and Invited Discussants
123
